A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease

Completed

Conditions studied: Pompe Disease, Glycogen Storage Disease Type II

In brief

The purpose of this study is to see how molecules called pharmacological chaperones affect the cells of patients with Pompe disease. The study will last 1 or 2 visits which will include a blood collection, urine collection, and two skin biopsies. Information will also be collected from the medical records about disease history and diagnosis. Patients will not receive any study medication.

Key facts

Study ID

NCT00515398

Run by

Amicus Therapeutics

People needed

30

Starts

2007-08-01

Expected to finish

2008-01-01

Last updated by the study team

2008-06-05

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female of any age
• Confirmed diagnosis of Pompe disease (early or late-onset)
• Clinically stable
• Written informed consent by subject or legal representative

You may not qualify if…

• Other significant disease or otherwise unsuitable for the study, as determined by the investigator

Where it is running

• University of Florida — Gainesville, Florida, United States
• Washington University — St Louis, Missouri, United States
• Center for Metabolic Disorders — Lake Success, New York, United States
• Duke University — Durham, North Carolina, United States

Full record on ClinicalTrials.gov

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